aTyr Pharma (ATYR) investor relations material

Platform and pipeline overview

• Focus on tRNA synthetase biology, advancing efzofitimod for pulmonary sarcoidosis and other interstitial lung diseases.

• Efzofitimod is a novel immunomodulator, now in phase III, with a unique mechanism validated in top-tier journals.

• Additional pipeline includes ATYR0101 for fibrosis and a small trial in scleroderma ILD, targeting unmet needs.

• Strong intellectual property position and approximately $70 million in cash support ongoing development.

Disease background and unmet need

• Pulmonary sarcoidosis is a serious orphan disease affecting nearly 200,000 in the U.S., with higher prevalence in African Americans.

• Current therapies rely on steroids and immunosuppressants, which are toxic and impair quality of life.

• Efzofitimod is the first therapy in 70 years to show improvement in key symptoms like cough, breathlessness, and fatigue.

• Large commercial opportunity estimated at $5 billion, with potential expansion to other interstitial lung diseases.

Clinical trial results and learnings

• EFZO-FIT phase III trial was the first to aggressively taper steroids, aiming to replace them with efzofitimod.

• While the primary endpoint (steroid reduction) was not statistically significant due to unexpectedly high placebo response, the trial changed global treatment guidelines.

• Significant and durable improvements in quality of life measures (cough, breathlessness, fatigue) were observed from week 8 onward.

• Safety profile remains favorable, with mostly mild to moderate adverse events and no new safety signals.